The Comparativeness Question - Drug Discovery and Development

A diverse coalition of lawmakers, industry representatives, healthcare providers, and patient advocacy groups is pressing the Obama Administration and Congress to make sure that newly authorized government comparativeness effectiveness research (CER) does not result in blocked Medicare payments for drugs or restricted patient access to needed treatments in the interest of cost-effectiveness.

The $787-billion economic stimulus plan passed earlier this year allocates $1.1 billion for CER at the National Institutes of Health (NIH), the Agency for Healthcare Research and Quality (AHRQ), and elsewhere in the government to “compare treatments and strategies to improve health.”

While generally supportive of CER, the pharmaceutical and biotech industries and many patient advocacy and physician groups fear the effort could lead down a slippery slope where potentially life-saving treatments would be stifled over cost concerns—the first step, they say, toward government health care rationing.

Canada and the UK use national agencies to conduct cost-effectiveness studies on drugs to determine their eligibility for and amount of government reimbursement. The UK’s National Institute for Health and Clinical Excellence (NICE) has been criticized in recent years for refusing to pay for new and potentially life-saving treatments that cost more than $45,000 annually.

The Obama Administration and Democratic lawmakers have made CER a key part of efforts to overhaul the nation’s health care system. While they have not drawn a direct connection to cost savings, the end result of CER, critics say, is simple: drugs and treatments that are not as effective or cost more than others would not be reimbursed to the same extent. This path leads to a government-run, single-payer health care system, warned Sen. Charles Grassley (R-Ia.), the top Republican on the Senate Finance Committee and a key player in health care reform.

“Here in the United States, only two people should be involved in deciding what treatment, drug, or device to use,” Grassley said in a speech on the Senate floor in March. “And these two people would be, on the one hand, the doctor; on the other hand, the patient.”

In March, the Department of Health and Human Services established a Coordinating Council for Comparative Effectiveness Research to oversee the $1.1-billion CER effort. Since then, the 15-member panel has held “listening sessions” in Chicago and Washington, D.C. to collect public comment before embarking on program recommendations. And in June, the Institute of Medicine issued a report recommending CER studies on 100 high-priority topics, including specific diseases and conditions, research methods and health care models. Final selection decisions are pending.

But some lawmakers, industry, and patient representatives are not waiting.

In May, Rep. Kurt Schrader (D-Ore.) introduced a bill (HR 2502) that would remove CER from the federal government and place it in the hands of an independent, non-governmental, nonprofit Health Care Comparative Effectiveness Research Institute. The legislation also bars the government from “mandating coverage, reimbursement, or other policies for any public or private payer.”

Among other things, the new institute would identify national priorities for CER, establish a committee to develop the science of CER, and disseminate research findings. All research would be peer-reviewed and those involved would be required to disclose conflicts of interest.

To make sure CER findings do not restrict access to drugs or other treatments, the bill includes a “physician out” clause: No CER reports or findings “shall be construed to prevent the physician and patient to ultimately determine what is best for the patient involved given the individual circumstances of different patients.”

The bill, called the Comparative Effectiveness Research Act, “will bring patients, along with health care providers, physicians, and industry to the decision-making process,” Schrader said in a letter to colleagues. “By having a seat at the table, the American people will help drive the direction of research based on what is more important to them.”

Members of the New Democratic Coalition, a group of moderate congressional Democrats, endorsed and cosponsored Schrader’s bill, which has been referred to two committees for consideration but not otherwise acted upon as of press time.

The bill also was endorsed by the Partnership to Improve Patient Care (PIPC), a 40-member group formed last November to support patient/provider-centric CER proposals. PIPC members include the Pharmaceutical Research and Manufacturers of America (PhRMA) and Biotechnology Industry Organization (BIO) trade organizations, and dozens of physician, patient, and advocacy associations.

The House bill is modeled after Senate legislation introduced last year by Democrats Max Baucus of Montana and Kent Conrad of North Dakota. In June, Baucus and Conrad, who now chair the powerful Finance and Budget committees, respectively, re-introduced the measure, called “The Patient-Centered Outcomes Research Act.”

Scott Gottlieb, MD a former senior US Food and Drug Administration (FDA) official during the George W. Bush Administration argues that the kinds of studies contemplated by the Obama Administration’s CER institute “fall short of the rigor that the FDA requires to support medical product approvals.”

Writing in the June “Health Policy Outlook,” a publication of the American Enterprise Institute where he is a resident fellow, Gottlieb and co-author Coleen Klasmeier, an attorney with Sidley Austin LLP in Washington, DC, call for establishing common standards for substantiating CER.

Without such standards, they say, the risk is that the government will become the sole arbiter of CER, since it alone would be exempt from FDA regulation while companies, held to higher standards, “might not only be prohibited from sharing similar CER, but also be unable to comment on the results of the studies generated by the Federal agency.”

Given the extent of interest in CER legislation expressed by moderate and mainline Democrats, however, it seems likely that the alternate proposals will be at least considered as the White House and congressional leaders craft health care reform measures.

About the Author
Contributing editor, Ted Agres, MBA, is a veteran science writer in Washington, DC. He writes frequently about the policy, politics, and business aspects of life sciences.